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(11)

EP 3 807 413 A1

(12)

(43) Date of publication: 21.04.2021 Bulletin 2021/16 (21) Application number: 19749864.5 (22) Date of filing: 17.06.2019

(51) International Patent Classification (IPC):  C12N 15/86(2006.01) A61K 48/00(2006.01) (86) International application number: PCT/US2019/037489 (87) International publication number: WO 2019/245973 (26.12.2019 Gazette 2019/52)

(84) Designated Contracting States: AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC MK MT NL NO PL PT RO RS SE SI SK SM TR Designated Extension States: BA ME Designated Validation States: KH MA MD TN (30) Priority: 18.06.2018 US 201862686668 P 02.10.2018 US 201862740402 P 30.10.2018 US 201862752841 P 25.03.2019 US 201962823649 P 11.06.2019 US 201962860220 P (71) Applicant: Research Institute at Nationwide Children's Hospital Columbus, Ohio 43205 (US)

(72) Inventors: RODINO-KLAPAC, Louise Groveport, OH 43125 (US) MENDELL, Jerry, R. Columbus, OH 43235 (US) (74) Representative: D Young & Co LLP  120 Holborn London EC1N 2DY London EC1N 2DY (GB)